Results 211 to 220 of about 214,719 (303)

Adeno-associated virus delivery of anti-alpha toxin monoclonal antibodies confers protection against Staphylococcus aureus infections. [PDF]

PLoS Pathog
Hommes JW, Hughes ME, Cheung D, Petri B, Orthner LM, van der Linden TJ, Bardoel BW, Deo SK, Patel DT, Flannagan RS, Heinrichs DE, Wootton SK, Surewaard BGJ.   +12 more
europepmc   +1 more source

Five‐Year Outcomes With Delandistrogene Moxeparvovec in Patients With Duchenne Muscular Dystrophy: A Phase 1/2a Study

Muscle &Nerve, EarlyView.
ABSTRACT Aims We report 5‐year results from a phase 1/2a study of delandistrogene moxeparvovec, a recombinant adeno‐associated virus serotype rh74 vector‐based gene therapy for Duchenne muscular dystrophy (DMD), with post hoc analyses contextualizing functional outcomes. Methods Four ambulatory patients with DMD (≥ 4–< 8 years at enrollment) entered an
Jerry R. Mendell, Zarife Sahenk, Linda P. Lowes, Megan A. Iammarino, Lindsay N. Alfano, Craig M. McDonald, James E. Signorovitch, Jim Jin, Jing Li, Stefanie Mason, Louise R. Rodino‐Klapac   +10 more
wiley   +1 more source

Pervasive horizontal transfer of adeno-associated virus capsid genes. [PDF]

Proc Natl Acad Sci U S A
Gifford RJ.
europepmc   +1 more source

Prime editing in neuropsychiatric disorders: From mutation‐specific target selection to clinical translation

Neuroprotection, EarlyView.
Abstract Prime editing, a novel clustered regularly interspaced short palindromic repeats (CRISPR)‐based technology, fuses a reverse transcriptase (RT) to an engineered CRISPR‐associated protein 9 (Cas9) and uses a prime editing guide RNA (pegRNA)‐encoded template.
Tianshan Ji, Yuan Zhang, Jinyi Zhao, Yiping Lu, Chengkun Wang   +4 more
wiley   +1 more source

Intranasal Vaccination with a Recombinant Adeno-Associated Virus Type 6 Encoding SapM Confers Protection Against Tuberculosis. [PDF]

Vaccines (Basel)
Xing C, Wang W, Yang J, Feng S, Xiao J, Cai N, Mo S, Cai Y, Chen X, Shi C.   +9 more
europepmc   +1 more source

The DNA/RNA autophagy protein SIDT2 as a novel neuropathological hallmark in Huntington disease

Brain Pathology, EarlyView.
SIDT2‐immunoreactive inclusions are observed in the striatum, cerebral cortex, and hypothalamus in HD cases with different Vonsattel grades, and the frequency of SIDT2‐immunoreactive inclusions is associated with longer CAG repeats in the huntingtin gene.
Sanaz Gabery, Sofia Bergh, Chrisovalantou Huridou, Rachel Y. Cheong, Barbara Baldo, Paul Günther Scheunemann, Marie‐Louisa Schoebel, Linda Holmquist Mengelbier, Elisabet Englund, Catriona McLean, Carsten Saft, Deniz Kirik, Maria Björkqvist, Glenda Halliday, Elisabeth Petrasch‐Parwez, Huu Phuc Nguyen, Jonasz Jeremiasz Weber, Åsa Petersén   +17 more
wiley   +1 more source

Classification and quantification of full vs. empty adeno-associated virus reference capsids via infrared attenuated total reflection spectroscopy. [PDF]

Anal Bioanal Chem
Lehner S, Stach R, Nilson R, Kochanek S, Kritzinger A, Sobek H, Kokoric V, Mizaikoff B.   +7 more
europepmc   +1 more source

PDE4D and PDE3B orchestrate distinct cAMP microdomains in 3T3‐L1 adipocytes

British Journal of Pharmacology, EarlyView.
Basal conditions: •Ins/PDE3B lowers cytoplasmic cAMP (cyt‐cAMP) without affecting plasma membrane cAMP (pm‐cAMP). •Insulin decreases lipid droplet cAMP (LD‐cAMP) independent of PDE3B. •FGF1/PDE4D modestly reduces both cyt‐ and pm‐cAMP, while PDE4D alone can modulate LD‐cAMP. ISO stimulation: •Ins/PDE3B has minimal impact on cyt‐cAMP.
Johannes Krier, David Spähn, David Arturo Juarez Lopez, Judith L. Nono, Judith Seigner, Lisa Jacob, Siegfried Ussar, Robert Lukowski, Andreas L. Birkenfeld, Gencer Sancar   +9 more
wiley   +1 more source

Consequences of human genetic variations in <i>KIAA0319L</i>, encoding adeno-associated virus receptor, on AAV-mediated gene transfer. [PDF]

Mol Ther Adv
Vasquez JM, Mackey HL, Rostami MR, Leopold PL, Crystal RG.   +4 more
europepmc   +1 more source

An intracellular recombinant single‐chain variable antibody fragment as a new class of phosphodiesterase type 5 inhibitors

British Journal of Pharmacology, EarlyView.
Background and Purpose Cyclic guanosine monophosphate (cGMP) is a ubiquitous second messenger involved in human (patho‐)physiology. Phosphodiesterase 5 (PDE5) is a major cGMP hydrolyzing enzyme in many cell types including vascular smooth muscle cells (VSMCs). Several highly selective PDE5 inhibitors are in clinical use. However, there are currently no
Kürsat Kirkgöz, Sophie Sprenger, Olga Schweigert, Sanika Mohagaonkar, Matti Walsdorff, Tanja Zeller, Filip Berisha, Viacheslav O. Nikolaev, Sergei D. Rybalkin   +8 more
wiley   +1 more source